Cadaveric Islet Transplantation in Patients With Insulin-Dependent Diabetes Mellitus
Primary Purpose
Islets of Langerhans Transplantation, Diabetes Mellitus, Type 1
Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Islets of Langerhans
Sponsored by
About this trial
This is an interventional treatment trial for Islets of Langerhans Transplantation focused on measuring islet transplantation, type 1 diabetes mellitus, steroid-free immunosuppression, rabbit anti-thymocyte globulin
Eligibility Criteria
Inclusion Criteria:
Group 1: Diagnosis of Type-1 diabetes mellitus for more than 5 years with at least one of the following complications:
- Metabolic lability/instability (two or more episodes of severe hypoglycemia) or two or more hospital visits for diabetic ketoacidosis during the previous year
- Progression of secondary complications of diabetes as determined by The Nebraska Medical Center/University of Nebraska Medical Center staff endocrinologists
- Group 2: Diagnosis of Type-1 diabetes with successful renal transplant on steroid-free, FK506/rapamycin-based immunosuppression
Exclusion Criteria:
- Severe co-existing cardiac disease
- Active alcohol or substance abuse, including cigarette smoking
- Psychiatric disorder making the subject not a suitable candidate for transplantation
- History of medical non-compliance
- Active infection, including hepatitis C and B, HIV, and tuberculosis (or suspected tuberculosis)
- Any history of malignancy except squamous or basal cell skin cancer
- BMI >28 kg/meter-squared, or body weight >80kg at screening visit, or >85kg on the day of transplantation (due to the difficulty of obtaining a sufficiently large islet mass to adequately treat either large patients or those whose obesity elevates their insulin needs)
- Positive C-peptide response to intravenous glucose tolerance test and Mixed Meal glucose tolerance test: any C-peptide >0.3 ng/mL post infusion
- Inability to provide informed consent
- Age less than 19 or greater than 70 years
- Creatinine clearance <60 mL/min/1.73 meter-squared for Group 1 and creatinine clearance <40 mL/min/1.73 meter-squared for Group 2 (those subjects currently on immunosuppression due to previous kidney transplant)
- Macroalbuminuria (urinary albumin excretion rate >300 mg/24h) for Group 1 and macroalbuminuria (urinary albumin excretion rate >600mg/24h) for Group 2
- Baseline Hb <10 gm/dL
- Baseline liver function tests outside of normal range
- Presence of gallstones or hemangioma in liver on baseline ultrasound exam
- Positive pregnancy test, intention of future pregnancy, or presently breast-feeding
- Evidence of sensitization on PRA
- Insulin requirement >0.7 IU/kg/day or HbA1c >15%
- Hyperlipidemia
- Under treatment for a medical condition requiring chronic use of steroids
- Use of Coumadin or other anticoagulant therapy (except aspirin) or PT-INR>1.5
- Diagnosis of Addison's disease
Additional Exclusion Criteria for Group 2 Subjects:
- Any history of organ transplantation other than kidney or pancreas
- Any previous graft lost to rejection
- Any history of early, multiple, or vascular renal allograft rejection
Sites / Locations
- The Nebraska Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Transplantation of Islets of Langerhans
Arm Description
Subjects will receive islets isolated from one donor pancreas per transplantation event. Subjects will receive a cumulative dose of 8,000 International Units/kg. Islets will be infused intraportally. If necessary, additional transplantation events will be performed.
Outcomes
Primary Outcome Measures
Incidence of insulin independence with a single islet transplant
Incidence of insulin independence with a single transplantation measured at one year post-transplantation.
Secondary Outcome Measures
Islet mass resulting in insulin independence/reduced exogenous insulin requirement
Insulin independence or reduced exogenous insulin required measured at one year post-transplantation.
Graft survival
The survival of the Islets of Langerhans graft as measured at three years post-transplantation.
Metabolic functional assessments of the islet graft
An assessment of the metabolic function of the islet of Langerhans graft as measured three years post-transplantation.
Full Information
NCT ID
NCT00579371
First Posted
December 17, 2007
Last Updated
July 12, 2023
Sponsor
University of Nebraska
1. Study Identification
Unique Protocol Identification Number
NCT00579371
Brief Title
Cadaveric Islet Transplantation in Patients With Insulin-Dependent Diabetes Mellitus
Official Title
Cadaveric Islet Transplantation in Patients With Insulin-Dependent Diabetes Mellitus
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Terminated
Why Stopped
Per Coordinator: study was closed and data was never analyzed
Study Start Date
March 17, 2004 (Actual)
Primary Completion Date
March 2011 (Actual)
Study Completion Date
March 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Nebraska
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
We hypothesize that the following improvements to islet transplantation will increase the islet mass successfully isolated and allow for engraftment from a single pancreas. The improvements are:
Using Two-Layer method preservation to improve pancreas quality before islet isolation
Maintaining isolated islets in culture before transplantation
Using a steroid-free immunosuppression regimen
Transplanting the best combination of donor and recipient possible after human leukocyte antigens (HLA) screening and final crossmatching
Detailed Description
We hypothesize that the following improvements to islet transplantation will increase the islet mass successfully isolated and allow for engraftment from a single pancreas:
using Two-Layer method preservation to improve pancreas quality before islet isolation, maintaining isolated islets in culture before transplantation, using a steroid-free immunosuppression regimen, and transplanting the best combination of donor and recipient possible after HLA screening and final crossmatching.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Islets of Langerhans Transplantation, Diabetes Mellitus, Type 1
Keywords
islet transplantation, type 1 diabetes mellitus, steroid-free immunosuppression, rabbit anti-thymocyte globulin
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Transplantation of Islets of Langerhans
Arm Type
Experimental
Arm Description
Subjects will receive islets isolated from one donor pancreas per transplantation event. Subjects will receive a cumulative dose of 8,000 International Units/kg. Islets will be infused intraportally. If necessary, additional transplantation events will be performed.
Intervention Type
Drug
Intervention Name(s)
Islets of Langerhans
Other Intervention Name(s)
transplanted islets of Langerhans
Intervention Description
Subjects will receive islets isolated from one donor pancreas per transplantation event. Subjects will receive a cumulative dose of 8,000IE/kg. Islets will be infused intraportally. If necessary, additional transplantation events will be performed.
Primary Outcome Measure Information:
Title
Incidence of insulin independence with a single islet transplant
Description
Incidence of insulin independence with a single transplantation measured at one year post-transplantation.
Time Frame
1 year post-transplantation
Secondary Outcome Measure Information:
Title
Islet mass resulting in insulin independence/reduced exogenous insulin requirement
Description
Insulin independence or reduced exogenous insulin required measured at one year post-transplantation.
Time Frame
1 year post-transplantation
Title
Graft survival
Description
The survival of the Islets of Langerhans graft as measured at three years post-transplantation.
Time Frame
3 years post-transplantation
Title
Metabolic functional assessments of the islet graft
Description
An assessment of the metabolic function of the islet of Langerhans graft as measured three years post-transplantation.
Time Frame
3 years post-transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
19 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Group 1: Diagnosis of Type-1 diabetes mellitus for more than 5 years with at least one of the following complications:
Metabolic lability/instability (two or more episodes of severe hypoglycemia) or two or more hospital visits for diabetic ketoacidosis during the previous year
Progression of secondary complications of diabetes as determined by The Nebraska Medical Center/University of Nebraska Medical Center staff endocrinologists
Group 2: Diagnosis of Type-1 diabetes with successful renal transplant on steroid-free, FK506/rapamycin-based immunosuppression
Exclusion Criteria:
Severe co-existing cardiac disease
Active alcohol or substance abuse, including cigarette smoking
Psychiatric disorder making the subject not a suitable candidate for transplantation
History of medical non-compliance
Active infection, including hepatitis C and B, HIV, and tuberculosis (or suspected tuberculosis)
Any history of malignancy except squamous or basal cell skin cancer
BMI >28 kg/meter-squared, or body weight >80kg at screening visit, or >85kg on the day of transplantation (due to the difficulty of obtaining a sufficiently large islet mass to adequately treat either large patients or those whose obesity elevates their insulin needs)
Positive C-peptide response to intravenous glucose tolerance test and Mixed Meal glucose tolerance test: any C-peptide >0.3 ng/mL post infusion
Inability to provide informed consent
Age less than 19 or greater than 70 years
Creatinine clearance <60 mL/min/1.73 meter-squared for Group 1 and creatinine clearance <40 mL/min/1.73 meter-squared for Group 2 (those subjects currently on immunosuppression due to previous kidney transplant)
Macroalbuminuria (urinary albumin excretion rate >300 mg/24h) for Group 1 and macroalbuminuria (urinary albumin excretion rate >600mg/24h) for Group 2
Baseline Hb <10 gm/dL
Baseline liver function tests outside of normal range
Presence of gallstones or hemangioma in liver on baseline ultrasound exam
Positive pregnancy test, intention of future pregnancy, or presently breast-feeding
Evidence of sensitization on panel reactive antibodies (PRA)
Insulin requirement >0.7 IU/kg/day or HbA1c >15%
Hyperlipidemia
Under treatment for a medical condition requiring chronic use of steroids
Use of Coumadin or other anticoagulant therapy (except aspirin) or a prothrombin time/international normalized ratio (PT-INR) >1.5
Diagnosis of Addison's disease
Additional Exclusion Criteria for Group 2 Subjects:
Any history of organ transplantation other than kidney or pancreas
Any previous graft lost to rejection
Any history of early, multiple, or vascular renal allograft rejection
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
R Brian Stevens, MD PhD
Organizational Affiliation
University of Nebraska
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
12. IPD Sharing Statement
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Cadaveric Islet Transplantation in Patients With Insulin-Dependent Diabetes Mellitus
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